Two cases of probable Neuro-Behçet syndrome treated with autologous HSCT

Neuro-Behçet syndrome (NBS) is a rare but detrimental neurological manifestation of the Behçet syndrome (BS) - a chronic multisystemic inflammatory disease. NBS is frequently associated with brainstem and spinal cord lesions, often resulting in substantial neurological disability. Atypical clinical presentations can pose significant challenges for diagnosis and treatment of NBS. Here, we report the clinical course, treatment strategies and responses of two cases with probable NBS. Both cases presented with longitudinally extensive transverse myelitis and brainstem lesions and experienced recurrent relapses as well as disease progression resistant to multiple immunotherapies. Autologous hematopoietic stem cell transplantation (aHSCT) was without beneficial effect on the disease course in both cases. In summary, NBS or probable NBS should be considered in patients with myelitis and brainstem involvement with limited treatment response. These cases illustrate diagnostic challenges and limitations of current diagnostic criteria, while also underscoring the heterogeneity of disease presentation. Immune reset by aHSCT failed to confer clinical benefit as a rescue therapy in these two patients. Further studies are required to optimize therapeutic strategies for NBS.